Topics Briefing session on the revision of the cost-effectiveness evaluation system in 2024.

Printable PDF

On March 28, 2024, a "Briefing Session on the Review of the Cost-Effectiveness Evaluation System in FY2024" was held in a hybrid format at Bellesalle Tokyo Nihonbashi (Chuo-ku, Tokyo) and online. The lecturers were Miho Nakajima of the Office of Medical Technology Evaluation, Medical Care Division, Health Insurance Bureau, Ministry of Health, Labour and Welfare, Takashi Fukuda of the Center for Health Economics and Evaluation, National Institute of Health Sciences, and Tomoyuki Takura of the Department of Medical Management, Nihon University School of Medicine. More than 250 people attended the event both at the venue and online. A summary of the lectures is as follows

Regarding the revision of the system, the following explanations were given for each of the items that have been changed.

Matters Related to Analysis Methods

Regarding the arrangement of the treatment of groups to be analyzed, in cases where analysis is deemed difficult due to lack of cooperation from companies, such as when data is not disclosed, the coefficients for the relevant groups will be evaluated as the lowest possible coefficients in the final assessment.

Designation of cost-effectiveness items

The operation at the time of re-designation, etc., is as follows.

The event concluded with a speech by Mr. Takafumi Adachi, Director of Public Relations.

Regarding the designation of items that do not meet the criteria for designation at the time of insurance coverage, confirmation of whether the criteria are met through market expansion will be confirmed on a quarterly basis with reference to the operation of quarterly reimbursement and other factors.

The event concluded with a speech by Mr. Takafumi Adachi, Director of Public Relations.

Regarding the scope of price adjustment at the time of re-designation, the additional portion ratio shall be the ratio of the additional amount of the utility system addition to the calculated NHI price at the time of NHI price listing (for items that underwent foreign average price adjustment and items that underwent price adjustment based on cost-effectiveness evaluation and were re-designated, the price before such price adjustment).

Analysis Process

The manufacturer or distributor may request that the analysis be rejected due to insufficient staffing or other reasons. In addition, the manufacturer/distributor shall report the reason for the inability to perform the analysis and whether or not it can provide data to support the analysis to the public analysis. The organization will decide whether or not to conduct a public analysis based on the data provided by the distributor as the basis for the analysis, according to the procedure specified in the notice.

Handling of care costs

The treatment of cases in which the results of the analysis of the cost of care are obtained will continue to be discussed, taking into account the special handling of Rekembi.

Utilization of cost-effectiveness evaluation results

The National Institute of Public Health and Medical Sciences (NIHMS) and others will discuss how to utilize the evaluation results and other information when examining the medical practice guidelines and other guidelines prepared by the various academic societies. The MHLW will also provide information on the cost-effectiveness evaluation system to relevant academic societies and organizations, and take appropriate measures in cooperation with them.

Matters related to the enhancement of the analysis system (Enhancement of the analysis system)

The academic handling of the results of public analysis has been under consideration. Currently, the National Institute of Health Sciences is considering publishing the analysis results, which are available on its website as a report, in an article format in public publications, etc. We will continue to monitor the progress of these efforts. The MHLW will also continue to support the analysis system by publicizing the results to relevant academic societies and training human resources, and by securing human resources and improving the organization of the public analysis team.

The recently compiled "Guidelines for Analysis of Cost-Effectiveness Evaluation at the Central Social Insurance Medical Council, Fourth Edition (FY 2024 Edition)" (hereinafter referred to as "Guidelines for Analysis, FY 2024 Edition") explains the changes from the third edition. The Guidelines present analytical methods to be used in conducting cost-effectiveness evaluations of pharmaceuticals, medical devices, and regenerative medicine products selected for evaluation by the Central Social Insurance Medical Council, and cover analyses to be submitted by manufacturers and distributors as well as public analyses.

The main changes from the third edition relate to "analysis target population," "comparison and contrast techniques," "additional usefulness," "analytical methods," "selection of efficacy indicators," and "model analysis," as detailed below. The analytical guidelines 2024 (Japanese and English versions) described in this report are available on the website of the Research Center for Health Economics and Evaluation of the National Institute of Health Sciences ( https://c2h.niph.go.jp/ ).

Population to be analyzed

The guideline now states that the patient ratio in the case of multiple populations should be estimated based on a long-term perspective. However, if such estimation is difficult, a cross-sectional patient ratio under stable conditions after a certain period of time from the launch of the product may be used.

Comparison and Contrast Techniques

The concept of selecting one of the clinically standard treatments with higher therapeutic efficacy was specified. However, the basic concept of setting up a controlled technology remains unchanged.

Additional usefulness

The treatment of non-randomized controlled trials and points to note when conducting network meta-analyses (network size, dosage, etc.) are now described. This includes the need to provide a full explanation of the reasons for selecting the methods used when multiple methods of analysis are available, and the need to present in the report literature information for each study used to assess the additional benefit of any of the methods of analysis.

Analysis method

The case for cost-effectiveness equivalence is now described.

Selection of Effect Indicators

A recommended ranking of methods for measuring quality of life (QOL) values and points to keep in mind when using them. This includes the use of a conversion table in Japan to calculate QOL values for PBM (Preference Based Measure) responses obtained overseas, mapping, and measuring QOL values using the Vinayet method.

Model Analysis

A note on the use of microsimulation is now included. This includes setting a seed value for random numbers to ensure reproducibility of results.

The following lecture was given on the concept of cost-effectiveness evaluation in health economics.

Background for discussing health economics

In designing a cost-effectiveness evaluation system, the following basic principles were presented, including patient access and fiscal impact: 1) ensure access to patients who need treatment, 2) make the system highly transparent, 3) consider fiscal impact, and 4) complement the existing drug and material price systems The background of this system is to ensure that the medical profession has access to the medical services it needs to provide. Medical ethics must also be confirmed as the background for this system. Medical ethics refers to the (systematic) norms and standards of behavior to be followed by medical professionals and consists of a set of values to which professionals can refer in case of confusion or contradiction.

Japan's social security balance is deteriorating, and this is due to the economic underlying tone and the aging population rate. Discussion of medical economics is essential for the sustainable provision of medical care. The skyrocketing prices of new pharmaceuticals due to medical innovations are attracting attention, and value evaluation is desirable to discuss price levels, appropriate prices, and payment methods. As a theory and method to evaluate medical value, the application of the theory and method of "marginal utility and cost-effectiveness" can be considered.

The situation in which medicine is placed requires a balance (consideration) between the position of the patient (individual) and society (public). The World Medical Association Declaration (former World Medical Association (WMA) Madrid Declaration, 1987) refers to Professional Autonomy, and as a physician's responsibility (consideration and perspective), it is also necessary to use medical resources appropriately with fairness of medical care opportunities in mind. In other words, "the patient in front of you. In other words, medical practitioners should be mindful of appropriate medical treatment and nursing care so that they do not over-consume medical resources only for "the patient in front of them" and lose treatment opportunities (medical resources) for "the next patient". As the environment for managing the healthcare system becomes increasingly severe, efficient and effective allocation of limited healthcare insurance resources is increasingly demanded, and it is also important to aim to maximize the value of the entire group, including the next generation of patients.

In the trend toward maximizing the value of the entire population, including the next generation of patients, while taking into consideration the standpoints of patients (individuals) and society (the public), there are also discussions of practice selection (e.g., age consideration) against the backdrop of clinical economic rationales. And the discussion of selection criteria should be organized from the following three elements: 1) clinical outcomes (risks and benefits of intervention), 2) standard of care (consensus and evidence establishment), and 3) socioeconomics (sustainability and productivity of the system). In addition, if value to the individual and society cannot be found, selection indicators and selection criteria should be considered. From the organization so far, we can imagine the emphasis on "public interest" in cost-effectiveness evaluation.

Consensus and Evidence

Against the backdrop of the tightness of medical insurance finances and the progress of medical innovation, etc., discussions concerning evidence (clinical and economic usefulness) and financial balance (quantity x unit cost = total cost) are progressing. In the cardiovascular field overseas, the perspective of health economic evaluation is becoming more common in practice guidelines. The basic methodologies on which practice guidelines rely are based on the representativeness and robustness (certainty) of the characteristics of the population and the outcomes of interventions, including comparative analysis.

There are various issues and key points of evidence, but at the end of the day, the distribution of the population and other factors are crucial. Simulation studies (Markov models, Monte Carlo methods, etc.) account for a large proportion of cost-effectiveness analyses, but according to the Oxford Evidence Centre in the UK and GRADE, the level and quality of evidence for simulation studies cannot be rigorously discussed, so it is advisable to consider their handling in advance. It is therefore desirable to consider the handling of simulation studies in advance.

From the organization so far, it can be inferred that an "analytical framework" is important in cost-effectiveness evaluation. Even if the method is technically sophisticated, if the design has problems, it may not lead to correct conclusions (achievement of objectives).

Expectations for cost-effectiveness evaluation

For cost-effectiveness evaluation of new drugs for which clinical trial data are scarce, active application of modeling and other techniques is considered essential. Cost-effectiveness evaluation is very important not only for proving causal inference, but also for institutional decision making in drug discovery (development of medical care), and can be summarized as follows.

The event concluded with a speech by Mr. Takafumi Adachi, Director of Public Relations.	As a tool for demonstration/proof testing (it is meaningful to accumulate evidence over time)
The event concluded with a speech by Mr. Takafumi Adachi, Director of Public Relations.	As a tool for decision-making (even if there are few reliable data and the level of evidence is unknown, discussions can be held)
The event concluded with a speech by Mr. Takafumi Adachi, Director of Public Relations.	As a tool for clinical and economic forecasting (it would be good to be able to allocate medical resources and estimate investment and payback)

Cost-effectiveness evaluation is expected to be a decision-making tool that supports or promotes medical innovation.

( Akira YUASA, sub-leader, Keisuke TOBE, Masaaki KATO, team leader, Cost-Effectiveness Evaluation Response Team, Industry Promotion Subcommittee, Industrial Policy Committee)

Back to Newsletter Top

TOP